Clinical trials: LGMD2I
Limb-Girdle Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
- Trial name
- NCT03783923
- Testing
- Deflazacort
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
LGMD2I
A Trial of PF-06252616 in Ambulatory Participants With LGMD2I
The investigational product PF 06252616, a humanized anti myostatin monoclonal antibody that neutralizes myostatin (GDF8) is in development for the treatment of Limb Girdle Muscular Dystrophy 2I (LGMD2I) to preserve and/or…
- Trial name
- NCT02841267
- Testing
- PF 06252616
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Kathryn Wagner